CA2998911A1 - Administration d'agents de potentialisation de cftr modifies au deuterium - Google Patents

Administration d'agents de potentialisation de cftr modifies au deuterium Download PDF

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Publication number
CA2998911A1
CA2998911A1 CA2998911A CA2998911A CA2998911A1 CA 2998911 A1 CA2998911 A1 CA 2998911A1 CA 2998911 A CA2998911 A CA 2998911A CA 2998911 A CA2998911 A CA 2998911A CA 2998911 A1 CA2998911 A1 CA 2998911A1
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Prior art keywords
compound
ivacaftor
ctp
pharmaceutically acceptable
administered
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CA2998911A
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CA2998911C (fr
Inventor
Virginia BRAMAN
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Vertex Pharmaceuticals Europe Ltd
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Vertex Pharmaceuticals Europe Ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/146—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic macromolecular compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2009—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2013—Organic compounds, e.g. phospholipids, fats
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2013—Organic compounds, e.g. phospholipids, fats
    • A61K9/2018—Sugars, or sugar alcohols, e.g. lactose, mannitol; Derivatives thereof, e.g. polysorbates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • A61K9/2022—Organic macromolecular compounds
    • A61K9/205—Polysaccharides, e.g. alginate, gums; Cyclodextrin
    • A61K9/2054—Cellulose; Cellulose derivatives, e.g. hydroxypropyl methylcellulose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/12—Mucolytics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D215/00—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/48—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
    • C07D215/54—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3
    • C07D215/56—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3 with oxygen atoms in position 4

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Pulmonology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

L'invention concerne une méthode de traitement de patients permettant de traiter chez le patient des maladies et états qui sont avantageusement traités par l'administration d'un agent de potentialisation du CFTR. La méthode comprend l'administration quotidienne au patient d'une quantité dans la plage comprise entre environ 50 mg et environ 200 mg du Composé (I) ou (II) ou de sels pharmaceutiquement acceptables de ces derniers. Cette invention concerne également des compositions comprenant le Composé (I) ou (II) et l'utilisation de telles compositions au sein de méthodes.
CA2998911A 2015-09-21 2016-09-21 Administration d'agents de potentialisation de cftr modifies au deuterium Active CA2998911C (fr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562221531P 2015-09-21 2015-09-21
US62/221,531 2015-09-21
US201562238511P 2015-10-07 2015-10-07
US62/238,511 2015-10-07
US201662348855P 2016-06-10 2016-06-10
US62/348,855 2016-06-10
PCT/US2016/052922 WO2017053455A1 (fr) 2015-09-21 2016-09-21 Administration d'agents de potentialisation de cftr modifiés au deutérium

Publications (2)

Publication Number Publication Date
CA2998911A1 true CA2998911A1 (fr) 2017-03-30
CA2998911C CA2998911C (fr) 2023-05-23

Family

ID=58387264

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2998911A Active CA2998911C (fr) 2015-09-21 2016-09-21 Administration d'agents de potentialisation de cftr modifies au deuterium

Country Status (16)

Country Link
US (2) US20180353500A1 (fr)
EP (2) EP4292588A3 (fr)
JP (2) JP6849686B2 (fr)
KR (1) KR20180058745A (fr)
CN (1) CN108367002A (fr)
AU (2) AU2016326441B2 (fr)
BR (1) BR112018005454A2 (fr)
CA (1) CA2998911C (fr)
HK (1) HK1258472A1 (fr)
IL (2) IL257993A (fr)
MA (1) MA42950A (fr)
MX (1) MX2018003331A (fr)
RU (1) RU2761344C2 (fr)
SG (1) SG10201913588YA (fr)
UA (1) UA124619C2 (fr)
WO (1) WO2017053455A1 (fr)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE047354T2 (hu) 2011-05-18 2020-04-28 Vertex Pharmaceuticals Europe Ltd Ivacaftor deuterizált származékai
SG10201913603QA (en) 2014-10-06 2020-02-27 Vertex Pharma Modulators of cystic fibrosis transmembrane conductance regulator
CA2981495C (fr) 2015-03-31 2023-09-26 Vertex Pharmaceuticals (Europe) Limited Vx-661 deutere
MX2018003331A (es) 2015-09-21 2018-08-16 Vertex Pharmaceuticals Europe Ltd Administracion de potenciadores de regulador de la conductancia transmembrana de fibrosis quistica (cftr) deuterados.
CA2999529A1 (fr) 2015-09-25 2017-03-30 Vertex Pharmaceuticals (Europe) Limited Potentialisateurs cftr deuteres
PL3436446T3 (pl) 2016-03-31 2023-09-11 Vertex Pharmaceuticals Incorporated Modulatory mukowiscydozowego przezbłonowego regulatora przewodnictwa
LT3519401T (lt) 2016-09-30 2021-11-25 Vertex Pharmaceuticals Incorporated Cistinės fibrozės transmembraninio laidumo reguliavimo moduliatorius, farmacinės kompozicijos, gydymo būdai ir moduliatoriaus gamybos būdas
US10793547B2 (en) 2016-12-09 2020-10-06 Vertex Pharmaceuticals Incorporated Modulator of the cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator
AU2018279646B2 (en) 2017-06-08 2023-04-06 Vertex Pharmaceuticals Incorporated Methods of treatment for cystic fibrosis
WO2019018353A1 (fr) * 2017-07-17 2019-01-24 Vertex Pharmaceuticals Incorporated Méthodes de traitement de la fibrose kystique
CA3069226A1 (fr) * 2017-07-17 2019-01-24 Vertex Pharmaceuticals Incorporated Methodes de traitement de la fibrose kystique
CN111051280B (zh) 2017-08-02 2023-12-22 弗特克斯药品有限公司 制备吡咯烷化合物的方法
CA3078893A1 (fr) 2017-10-19 2019-04-25 Vertex Pharmaceuticals Incorporated Formes cristallines et compositions de modulateurs de cftr
US11708331B2 (en) 2017-12-01 2023-07-25 Vertex Pharmaceuticals Incorporated Processes for making modulators of cystic fibrosis transmembrane conductance regulator
JP7245834B2 (ja) 2017-12-08 2023-03-24 バーテックス ファーマシューティカルズ インコーポレイテッド 嚢胞性線維症膜コンダクタンス制御因子のモジュレーターを作成するためのプロセス
TWI810243B (zh) 2018-02-05 2023-08-01 美商維泰克斯製藥公司 用於治療囊腫纖化症之醫藥組合物
PT3752510T (pt) 2018-02-15 2023-03-15 Vertex Pharma Macrociclos como moduladores do regulador de condutância de transmembrana da fibrose cística, suas composições farmacêuticas, seu uso no tratamento da fibrose cística e processos para produzi-los
US11414439B2 (en) 2018-04-13 2022-08-16 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator, pharmaceutical compositions, methods of treatment, and process for making the modulator
FI3880197T3 (fi) 2018-11-14 2023-05-04 Vertex Pharma Menetelmiä kystisen fibroosin hoitamiseksi
AR118555A1 (es) 2019-04-03 2021-10-20 Vertex Pharma Agentes moduladores del regulador de la conductancia transmembrana de la fibrosis quística
WO2020214921A1 (fr) 2019-04-17 2020-10-22 Vertex Pharmaceuticals Incorporated Formes solides de modulateurs de cftr
FR3096890B1 (fr) 2019-06-07 2021-05-14 H4 Orphan Pharma Utilisation d’une molécule opioïde pour traiter l’œil sec et l’œil allergique.
TW202115092A (zh) 2019-08-14 2021-04-16 美商維泰克斯製藥公司 囊腫纖維化跨膜傳導調節蛋白之調節劑
BR112022002606A2 (pt) 2019-08-14 2022-05-03 Vertex Pharma Moduladores do regulador de condutância transmembrana da fibrose cística
BR112022002605A2 (pt) 2019-08-14 2022-05-03 Vertex Pharma Formas cristalinas de moduladores de cftr
TW202120517A (zh) 2019-08-14 2021-06-01 美商維泰克斯製藥公司 製備cftr調節劑之方法
FR3109524B1 (fr) 2020-04-22 2022-04-08 H4 Orphan Pharma Utilisation d’un ligand multifonctionnel pour traiter l’œil sec et les dysfonctionnements des glandes de Meibomius et des glandes lacrymales.
FR3111071B1 (fr) 2020-06-04 2023-01-13 H4 Orphan Pharma Utilisation de ligands multifonctionnels pour traiter les syndromes de détresse respiratoire et le choc cytokinique liés aux infections virales à coronavirus.
CR20230120A (es) 2020-08-07 2023-09-01 Vertex Pharma Moduladores del regulador de la conductancia transmembrana de la fibrosis quística
JP2023537944A (ja) 2020-08-13 2023-09-06 バーテックス ファーマシューティカルズ インコーポレイテッド Ｃｆｔｒモジュレーターの結晶形態
WO2022076624A1 (fr) 2020-10-07 2022-04-14 Vertex Pharmaceuticals Incorporated Modulateurs du régulateur de conductance transmembranaire de la fibrose kystique
WO2022076618A1 (fr) 2020-10-07 2022-04-14 Vertex Pharmaceuticals Incorporated Modulateurs du régulateur de la conductance transmembranaire de la fibrose kystique
US20230373974A1 (en) 2020-10-07 2023-11-23 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
JP2023545080A (ja) 2020-10-07 2023-10-26 バーテックス ファーマシューティカルズ インコーポレイテッド 嚢胞性線維症膜コンダクタンス制御因子モジュレーター
US20230373939A1 (en) 2020-10-07 2023-11-23 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
EP4225763A1 (fr) 2020-10-07 2023-08-16 Vertex Pharmaceuticals Incorporated Modulateurs du régulateur de conductance transmembranaire de la fibrose kystique
CA3197173A1 (fr) 2020-10-07 2022-04-14 Vertex Pharmaceuticals Incorporated Modulateurs du regulateur de la conductance transmembranaire de la mucoviscidose
US20230373935A1 (en) 2020-10-07 2023-11-23 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
WO2022076621A1 (fr) 2020-10-07 2022-04-14 Vertex Pharmaceuticals Incorporated Modulateurs du régulateur de la conductance transmembranaire de la fibrose kystique
EP4259139A1 (fr) 2020-12-10 2023-10-18 Vertex Pharmaceuticals Incorporated Méthodes de traitement de la mucoviscidose
WO2023150237A1 (fr) 2022-02-03 2023-08-10 Vertex Pharmaceuticals Incorporated Méthodes de traitement de la fibrose kystique
WO2023154291A1 (fr) 2022-02-08 2023-08-17 Vertex Pharmaceuticals Incorporated Modulateurs du régulateur de la conductance transmembranaire de la fibrose kystique
AU2023249173A1 (en) 2022-04-06 2024-10-03 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
WO2023224931A1 (fr) 2022-05-16 2023-11-23 Vertex Pharmaceuticals Incorporated Méthodes de traitement de la fibrose kystique

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HUE049976T2 (hu) 2005-12-28 2020-11-30 Vertex Pharma N-[2,4-bisz(1,1-dimetil-etil)-5-hidroxi-fenil]-1,4-dihidro-4-oxo-kinolin-3-karboxamid amorf alakjának gyógyászati kompozíciói
JP5575768B2 (ja) 2008-08-13 2014-08-20 バーテックス ファーマシューティカルズ インコーポレイテッド 薬学的組成物およびその投与
AU2011255237A1 (en) * 2010-05-20 2012-11-29 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions and administrations thereof
HUE047354T2 (hu) * 2011-05-18 2020-04-28 Vertex Pharmaceuticals Europe Ltd Ivacaftor deuterizált származékai
RS59744B1 (sr) * 2011-05-18 2020-02-28 Vertex Pharmaceuticals Europe Ltd Deuterisani derivati ivakaftora
CN109966264A (zh) 2012-02-27 2019-07-05 沃泰克斯药物股份有限公司 药物组合物及其施用
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CN103833630B (zh) * 2012-11-21 2018-11-13 顶点制药（欧洲）有限公司 氘化cftr增效剂
US20140221424A1 (en) * 2013-01-30 2014-08-07 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions for use in the treatment of cystic fibrosis
MX2018003331A (es) 2015-09-21 2018-08-16 Vertex Pharmaceuticals Europe Ltd Administracion de potenciadores de regulador de la conductancia transmembrana de fibrosis quistica (cftr) deuterados.

Also Published As

Publication number Publication date
JP6849686B2 (ja) 2021-03-24
RU2018114447A3 (fr) 2020-02-21
CA2998911C (fr) 2023-05-23
AU2022201135A1 (en) 2022-03-17
EP3352757A1 (fr) 2018-08-01
RU2018114447A (ru) 2019-10-23
EP3352757A4 (fr) 2018-08-01
CN108367002A (zh) 2018-08-03
MX2018003331A (es) 2018-08-16
IL257993A (en) 2018-05-31
WO2017053455A1 (fr) 2017-03-30
JP7196217B2 (ja) 2022-12-26
BR112018005454A2 (pt) 2018-10-09
HK1258472A1 (zh) 2019-11-15
JP2018534348A (ja) 2018-11-22
SG10201913588YA (en) 2020-02-27
JP2021091723A (ja) 2021-06-17
KR20180058745A (ko) 2018-06-01
US20200375973A1 (en) 2020-12-03
AU2016326441A1 (en) 2018-04-19
IL291517A (en) 2022-05-01
EP3352757B1 (fr) 2023-08-16
US20180353500A1 (en) 2018-12-13
AU2022201135B2 (en) 2024-03-28
MA42950A (fr) 2018-08-01
EP4292588A3 (fr) 2024-02-28
AU2016326441B2 (en) 2021-11-25
RU2761344C2 (ru) 2021-12-07
EP3352757C0 (fr) 2023-08-16
EP4292588A2 (fr) 2023-12-20
UA124619C2 (uk) 2021-10-20

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